Inhaled therapy for Raynaud’s attacks put on FDA fast track

The U.S. Food and Drug Administration (FDA) has granted fast track designation to treprostinil, an inhaled therapy to treat Raynaud’s phenomenon — a cold, numb, or prickly sensation in the fingers and toes — associated with systemic sclerosis (SSc-RP).

The medication, approved under the brand name Yutrepia for the lung condition pulmonary arterial hypertension (PAH), is expected to widen blood vessels and improve blood flow in the fingers and toes, reducing the number and severity of Raynaud’s attacks.

The FDA’s fast track program is designed to support the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need. In this case, it provides Liquidia, the company that markets Yutrepia, with the opportunity for more frequent interactions with the agency throughout development. The drug may also be eligible for certain programs that speed its path to market, including rolling review, priority review and/or accelerated approval.

“Fast Track designation reflects the seriousness of this condition and the need for new options,” Rajeev Saggar, MD, chief medical officer at Liquidia, said in a company press release. “No therapy is currently approved by the FDA specifically for SSc-RP.”

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Clinical trial planned

The company also announced plans for a study assessing treprostinil in about 75 adults with SSc experiencing symptomatic Raynaud’s attacks. The RE-WARM Phase 2a clinical trial (NCT07748000) is expected to begin in October, with completion targeted for February 2027. Information on study locations has yet to be disclosed.

“We are encouraged by the opportunity to evaluate whether [treprostinil] can reduce the complications of moderate to severe Raynaud’s that most affect patients’ quality of life,” Saggar said.

SSc, also known as scleroderma, is an autoimmune disease characterized by widespread fibrosis (scarring) that can affect the skin, internal organs, and blood vessels.

Excessive buildup of scar tissue narrows small blood vessels and limits blood flow in the fingers and toes, causing Raynaud’s phenomenon, a condition in which the fingers and toes feel cold, numb, and prickly in response to cold temperatures or stress. Attacks affect up to 90% of SSc patients and often progress to ulcers and, in recurrent cases, finger/toe amputation.

SSc patients have identified Raynaud’s attacks as one of the disease’s most bothersome symptoms, citing pain, functional limitation, and emotional distress due to fear of ulcers and tissue loss, Liquidia noted.

A central driver of SSc-RP is a deficiency of prostacyclin, a naturally occurring hormone that helps blood vessels relax and widen, improving blood flow. Treprostinil is a lab-made formulation of prostacyclin. Liquidia’s approach delivers it through the lungs using an easy-to-use, palm-sized device.

The therapy is approved to improve exercise capacity in people with PAH, a condition marked by narrowing of the blood vessels that pass through the lungs. It’s also available for those with pulmonary hypertension associated with interstitial lung disease (PH-ILD), in which scarring and inflammation in the lungs raise blood pressure in the pulmonary blood vessels.

RE-WARM’s main goals are to test safety and to identify tolerated doses of treprostinil in adults with SSc who have experienced at least 12 symptomatic Raynaud’s attacks on at least four separate days. The study, expected to take place at around 30 sites across the U.S., will also explore whether treatment reduces the number, severity, and impact of Raynaud’s attacks.

We are committed to advancing this program with urgency, starting with the RE-WARM study later this year,” Saggar said.